Novartis unveils investigation of infant's death in European Zolgensma trial

Novartis
Novartis has launched an internal probe into the death of an infant in the EU trial for SMA gene therapy treatment Zolgensma. (Novartis)

On the cusp of Zolgensma's landmark FDA approval, Novartis has big hopes for the first-ever gene therapy for spinal muscular atrophy. But a patient’s death in the treatment's European trial could throw a wrench into its plans.

Novartis is digging into the death of an infant, reported by FiercePharma last Wednesday, who was treated as part of Zolgensma’s ongoing European phase 3 trial. Zolgensma, a one-time therapy targeting SMA Type 1 with a prospective price tag of up to $5 million, is poised for an FDA decision next month.

The infant's death was attributed to a severe respiratory infection and neurological complications. A trial investigator said the death might have been treatment-related, but autopsy results are pending, a spokesperson told FiercePharma. The company has shared the details with regulatory authorities.

Webinar This Week

OTC Innovation to Avoid Stagnation: Survey Insights, Expert Advice, and Latest Technologies to Boost Your Product’s Performance

Join us for a complimentary webinar on November 13 at 11am ET / 8am PT. Listen to industry experts as they analyze the critical role of innovation in OTC products, and strategies for achieving it.

The EU trial is still enrolling patients and none of its data have been reported yet. "In an effort to be transparent, we reported that this death occurred,” Novartis spokesman Eric Althoff  told FiercePharma Monday.

Novartis had disclosed the death of another Zolgensma patient in data released last Tuesday from a phase 3 trial dubbed STR1VE. In that study, 21 of 22 treated infants with SMA Type 1 were reported alive and free from serious complications at 9.5 months of age. Fifty percent of infants with untreated SMA Type 1 die or require ventilation by 10.5 months of age, Novartis said.

RELATED: Novartis beefs up its case for Zolgensma gene therapy ahead of FDA decision

The sole death in that trial wasn't related to treatment, according to an internal investigator and an independent Data Safety Monitoring Board, Novartis said.

In its bid to become the first approved gene therapy to treat SMA, Zolgensma—and its steep suggested price tag—is aiming to challenge Biogen’s Spinraza, which has zoomed to blockbuster status since its December 2016 approval. Spinraza, a once-a-trimester treatment for adults and infants, raked in $1.7 billion in worldwide sales in 2018.

RELATED: Biogen, Novartis need to think smaller—much smaller—on SMA drug prices: ICER

Both Spinraza and Zolgensma have been targeted by drug-price watchdogs for their eye-popping costs in recent months, with analysts saying the treatments’ prices outweigh their benefit to patients.

In February, the Institute for Clinical and Economic Research (ICER) proposed a $900,000 price tag for Zolgensma. Novartis said the one-time treatment could be cost-effective at $4 million to $5 million, but analysts figure the company will set a price closer to $2 million.

RELATED: NICE cost watchdogs spurn Biogen's pricey Spinraza but leave door open for a deal

For Spinraza, ICER suggested an aggressive price cut of around 83%, significantly lowering its first-year cost of $750,000 and $350,000 for each subsequent year.

Spinraza was previously knocked by England’s National Institute for Health and Care Excellence (NICE) for its price. In August 2018, analysts refused to recommend the treatment for listing on England’s NHS, saying Spinraza’s steep cost was not cost-effective in the long term despite its “substantial benefit” to patients with SMA.

Suggested Articles

Intercept presented a data analysis that found treatment with Ocaliva led to "early and consistent improvements" in a range of noninvasive tests.

Days before Amarin faces a pivotal FDA vote on its Vascepa expansion, advisors are set to scrutinize the placebo used in its pivotal outcomes trial.

Both IL-17A inhibitors have rolled out data showing they work in non-radiographic axial spondyloarthritis.